Overview of New Drugs Approved by the U.S. FDA in Q1 2025
Below is a list of new drugs approved by the U.S. FDA during the first quarter (January to March) of 2025, covering oncology, cardiovascular diseases, infectious diseases, and other fields, sorted by approval date:
1.Tabelecleucel (tab-cel)
Approval Date: January 15, 2025
Mechanism/Effect: An allogeneic, “off-the-shelf” EBV-specific T-cell immunotherapy targeting and eliminating EBV-infected cells for the treatment of Epstein-Barr virus-positive (EBV+) post-transplant lymphoproliferative disorder (PTLD).
Indication: EBV+ PTLD.
2.Datopotamab Deruxtecan (Dato-DXd)
Approval Date: January 17, 2025 (initially approved for unresectable or metastatic HR-positive, HER2-negative breast cancer in adults; pending decision for NSCLC).
Mechanism/Effect: A Trop2-targeted antibody-drug conjugate (ADC) for treating unresectable or metastatic hormone receptor-positive (HR+), HER2-negative breast cancer. Also granted FDA Priority Review for locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC).
Indication: Unresectable or metastatic HR+/HER2- breast cancer.
3.Treosulfan (Grafapex)
Approval Date: January 22, 2025
Mechanism/Effect: A conditioning regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in patients aged ≥1 year with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS).
Indication: Preparative therapy for MDS/AML patients undergoing stem cell transplantation.
4.VX-548 (Suzetrigine)
Approval Date: January 30, 2025
Mechanism/Effect: An oral selective NaV1.8 inhibitor that blocks peripheral nerve pain signals, providing rapid analgesia for moderate-to-severe acute pain without opioid addiction risks. Demonstrated efficacy in Phase III trials.
Indication: Acute pain (e.g., postoperative pain).
5.AXS-07 (Meloxicam/Rizatriptan Combination)
Approval Date: January 31, 2025
Mechanism/Effect: Rapidly alleviates acute migraine attacks by inhibiting CGRP release and neuroinflammation. Phase III trials showed significant pain relief within 2 hours compared to placebo.
Indication: Acute treatment of migraine.
6.Mirdametinib (Gomekli)
Approval Date: February 2025
Mechanism/Effect: An oral MEK inhibitor for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN). Phase II trials reported objective response rates (ORR) of 41% in adults and 52% in children.
Indication: NF1-PN.
7.Vimseltinib (Romvimza)
Approval Date: February 2025
Mechanism/Effect: A CSF-1R inhibitor that blocks tumor-associated macrophage activity for inoperable tenosynovial giant cell tumor (TGCT). Phase III trials demonstrated an ORR of 40%.
Indication: Tenosynovial giant cell tumor.
8.Gepotidacin (Blujepa)
Approval Date: March 26, 2025
Mechanism/Effect: A novel oral antibiotic targeting bacterial DNA gyrase and topoisomerase IV for uncomplicated urinary tract infections (uUTIs). Phase III trials showed superiority to nitrofurantoin and represents the first new-mechanism antibiotic in 20 years.
Indication: Uncomplicated urinary tract infections (acute cystitis) and gonococcal infections, including multidrug-resistant strains.
9.Fitusiran (Qfitlia)
Approval Date: March 28, 2025
Mechanism/Effect: An siRNA therapy that inhibits antithrombin III synthesis to prevent bleeding in hemophilia patients. Requires only six subcutaneous injections annually and is the first broad-spectrum therapy for hemophilia A/B (including inhibitor-positive patients).
Indication: Hemophilia A/B.
Summary
Q1 2025 FDA approvals focused on oncology (e.g., ADCs, MEK inhibitors) and rare diseases (e.g., hemophilia, NF1-PN), alongside advancements in infectious diseases (antibiotics) and non-opioid pain management. Key innovations include CRISPR-CAR-T therapies and AI-driven drug development (e.g., ISM001-055).
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